Recruiting
Phase 1

Nicotinamide, Tetrahydrouridine, Decitabine

Sponsor:

EpiDestiny, Inc.

Code:

NCT04055818

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Nicotinamide

Study Details

Brief summary:

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

Conditions

Sickle Cell Disease

Study ID

NCT04055818

Start date

Jan 24, 2020

Status verified date

May, 2025

Completion date

Apr 30, 2027

Anticipated

Primary completion date

Oct 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Age 18 years or older.
  • Written informed consent provided by the subject before study entry.
  • Confirmed sickle cell disease (SCD) as determined by hemoglobin electrophoresis or liquid chromatography.
  • Subject is in his/her steady state and not having any acute complication due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome in the past 14 days).
  • Weight at least 40kg
  • Regular compliance with comprehensive care and previous therapy.
  • Symptomatic SCD is defined as having one of following, despite at least 6 months of hydroxyurea therapy, or refuse to take hydroxyurea for personal reasons: fetal hemoglobin <0.5 g/dL, or 3 or more pain episodes per year requiring parenteral narcotics, or 1 or more acute chest syndrome episodes, or Hemoglobin <9 g/dL and absolute reticulocyte count <250,000/mm3.

Exclusion Criteria:

  • Inability to give informed consent.
  • Experienced severe sepsis or septic shock within the previous 12 weeks.
  • Last HU dose was ingested within the previous 4 weeks.
  • Currently pregnant or breast-feeding.
  • Alanine Aminotransferase (ALT) ≥ 3 times the upper limit of normal or albumin <2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl.
  • Serum creatinine >2.9 mg/dL and calculated creatinine clearance <30 mL/min.
  • Platelet count >800 x 109/L.
  • Absolute neutrophil count <1.5 x 109/L.
  • Female of active childbearing potential who is unwilling to use at least one of the two following forms of birth control: (i) not having heterosexual sexual contact beginning at the screening visit and continuing until 4 weeks after the last dose of decitabine OR (ii) intrauterine device (IUD).
  • Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a female with child-bearing potential, beginning at the screening visit and continuing until 4 weeks after taking the last dose of THU and decitabine. This requirement applies also to males who have had a successful vasectomy.
  • Altered mental status or recurrent seizures requiring anti-seizure medications.
  • Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely.
  • Concurrent diagnosis of malignancy including known Myelodysplastic syndrome, leukemia, or an abnormal karyotype.
  • New York Heart Association (NYHA) class III/IV status.
  • Eastern Co-operative Oncology Group (ECOG) performance status ≥3.
  • Participant is on chronic transfusion therapy
  • Known history of illicit drug or alcohol abuse within the past 12 months.
  • Other experimental or investigational drug therapy in the past 28 days.
  • Taking l-glutamine within the last 28 days
  • Being positive for HIV infection

Study Design

Enrollment

20 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Nicotinamide

Oral Nicotinamide 1000 mg twice daily

experimental: THU Decitabine

Oral 250 mg THU and 5 mg decitabine Once per week

Interventions

Nicotinamide

Oral nicotinamide (Vitamin B3) alone compared to THU Decitabine combination

Primary outcome measure

  • Blood Hemoglobin [ Time Frame: 12 weeks ]

Central Contacts and Locations

Central contacts

Lani Krauz

312-413-0242LIgnacio@UIC.EDU

Locations

University of Illinois at Chicago College of Medicine

Recruiting

Chicago, Illinois, United States, 60612

Contacts

Lani Krauz

312-413-0242Llgnacio@UIC.EDU

More Information

Sponsor

EpiDestiny, Inc.

Last update posted

May 25, 2025

Last verified

May, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by EpiDestiny, Inc. on 2025-05-25.